This page compiles news and announcements about the PaVe-GT program produced by participating NIH Institutes.

2023
NIH Gene Therapy Team Reveals Its Path to FDA Orphan Drug and Rare Pediatric Disease Designations
2022
NCATS Receives FDA Rare Pediatric Disease Designation to Treat Propionic Acidemia
The U.S. Food and Drug Administration (FDA) has granted the National Institutes of Health’s National Center for Advancing Translational Sciences (NCATS) a Rare Pediatric Disease (RPD) designation for AAV9-hPCCA (NCATS-BL0746), an investigational gene therapy for the treatment of propionic acidemia…
NCATS Receives FDA Orphan Drug Designation to Treat Propionic Acidemia
The U.S. Food and Drug Administration (FDA) has granted the National Institutes of Health (NIH)’s National Center for Advancing Translational Sciences (NCATS) an orphan drug designation for AAV9-hPCCA (NCATS-BL0746), an investigational gene therapy for the treatment of propionic acidemia resulting…
2020
PaVe-GT: Collaborative NIH Effort Aimed at Creating a Gene Therapy Playbook, Making Rare Disease Treatments More Accessible
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Pave-GT Resources
Rare Pediatric Disease (RPD) Designation Request for AAV9-hPCCA

This pdf file contains the RPD designation request for AAV9-hPCCA (NCATSBL-0746) and associated communications between NCATS and FDA OOPD.

March 21, 2023
Orphan Drug Designation (ODD) Request for AAV9-hPCCA

This pdf file contains the ODD request for AAV9-hPCCA (NCATSBL-0746) and associated communications between NCATS and FDA OOPD.

March 21, 2023
Rare Pediatric Disease (RPD) Designation Request Template Read More
February 27, 2023
Orphan Drug Designation (ODD) Request Template Read More
February 27, 2023
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